Human Metabolic Enzymes Produced in Transfected Plants

New methods are needed to produce pharmaceutical quality proteins for therapeutic use in humans onMany heritable metabolic storage disorders of humans are potentially treatable by supplementation wiproduced enzymes. For example, Gaucher's disease is caused by genetic deficiencies in the enzyme glucan be successfully treated by enzyme replacement therapy. However, cost and availability of the enztreatment. Plants are relatively unexplored production hosts for cost-effective biosynthesis of humarecently developed transient expression vectors based on tobacco mosaic virus, we will examine the fcustom-designed proteins in plants using glucocerebrosidase as a test enzyme. These vectors allow boexpression and rapid cycles of testing and modification of the enzyme. If the enzyme produced in plaactivity toward the accumulating glucocerebroside, we can rapidly test experimental alterations to fstability, and targeting of the enzyme to lipid storing cells. The long-term objectives are to develcopious amounts of glucocerebrosidase and other human enzymes for routine therapeutic treatments. Uscustom peptide synthesis could be designed for individuals that may be intolerant to the wild type e